Try the modernized ClinicalTrials.gov beta website. Learn more about the modernization effort.
Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

Safety and Efficacy of Nonacog Beta Pegol (N9-GP) in Previously Untreated Patients With Haemophilia B (paradigm™6)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02141074
Recruitment Status : Active, not recruiting
First Posted : May 19, 2014
Last Update Posted : December 1, 2021
Sponsor:
Information provided by (Responsible Party):
Novo Nordisk A/S

Tracking Information
First Submitted Date  ICMJE March 28, 2014
First Posted Date  ICMJE May 19, 2014
Last Update Posted Date December 1, 2021
Actual Study Start Date  ICMJE July 2, 2014
Estimated Primary Completion Date October 30, 2022   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: January 23, 2018)
  • Incidence of inhibitory antibodies against coagulation factor IX (FIX) [ Time Frame: When minimum 20 previously untreated patients (PUPs) have reached at least 50 exposure days (EDs) (after approx. 48 months) ]
  • Incidence of inhibitory antibodies against coagulation factor IX (FIX) [ Time Frame: When minimum 40 PUPs have reached at least 100 EDs (after approx. 82 months) ]
  • Incidence of inhibitory antibodies against coagulation factor IX (FIX) [ Time Frame: At end of trial (after approx. 100 months) ]
Original Primary Outcome Measures  ICMJE
 (submitted: May 16, 2014)
  • Incidence of inhibitory antibodies against coagulation factor IX (FIX) [ Time Frame: When the first 20 previously untreated patients (PUPs) have reached at least 50 exposure days (EDs) (Expected to reach between 12 - 24 months) ]
  • Incidence of inhibitory antibodies against coagulation factor IX (FIX) [ Time Frame: When the first 40 PUPs have reached 100 EDs. (Expected to reach between 24 - 36 months) ]
  • Incidence of inhibitory antibodies against coagulation factor IX (FIX) [ Time Frame: Expected to reach between 24 - 72 months ]
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: January 23, 2018)
  • Number and frequency of adverse events [ Time Frame: When minimum 20 PUPs have reached at least 50 EDs (after approx. 48 months) ]
  • Number and frequency of serious adverse events [ Time Frame: When minimum 20 PUPs have reached at least 50 EDs (after approx. 48 months) ]
  • Number and frequency of Medical Events of Special Interest [ Time Frame: When minimum 20 PUPs have reached at least 50 EDs (after approx. 48 months) ]
  • Number of breakthrough bleeding episodes during prophylaxis (annualised bleeding rate) [ Time Frame: When minimum 20 PUPs have reached at least 50 EDs (after approx. 48 months) ]
  • Haemostatic effect by 4-point haemostatic response scale ("excellent", "good", "moderate" and "poor") [ Time Frame: When minimum 20 PUPs have reached at least 50 EDs (after approx. 48 months) ]
  • Number and frequency of adverse events [ Time Frame: When minimum 40 PUPs have reached at least 100 EDs (after approx. 82 months) ]
  • Number and frequency of adverse events [ Time Frame: At end of trial (after approx. 100 months) ]
  • Number and frequency of serious adverse events [ Time Frame: When minimum 40 PUPs have reached at least 100 EDs (after approx. 82 months) ]
  • Number and frequency of serious adverse events [ Time Frame: At end of trial (after approx. 100 months) ]
  • Number and frequency of Medical Events of Special Interest [ Time Frame: When minimum 40 PUPs have reached at least 100 EDs (after approx. 82 months) ]
  • Number and frequency of Medical Events of Special Interest [ Time Frame: At end of trial (after approx. 100 months) ]
  • Number of breakthrough bleeding episodes during prophylaxis (annualised bleeding rate) [ Time Frame: When minimum 40 PUPs have reached at least 100 EDs (after approx. 82 months) ]
  • Number of breakthrough bleeding episodes during prophylaxis (annualised bleeding rate) [ Time Frame: At end of trial (after approx. 100 months) ]
  • Haemostatic effect by 4-point haemostatic response scale ("excellent", "good", "moderate" and "poor") [ Time Frame: When minimum 40 PUPs have reached at least 100 EDs (after approx. 82 months) ]
  • Haemostatic effect by 4-point haemostatic response scale ("excellent", "good", "moderate" and "poor") [ Time Frame: At end of trial (after approx. 100 months) ]
Original Secondary Outcome Measures  ICMJE
 (submitted: May 16, 2014)
  • Number and frequency of adverse events [ Time Frame: When the first 20 PUPs have reached at least 50 EDs, when the first 40 PUPs have reached 100 EDs, and at end of trial (Expected to reach between 12 - 72 months) ]
  • Number and frequency of serious adverse events [ Time Frame: When the first 20 PUPs have reached at least 50 EDs, when the first 40 PUPs have reached 100 EDs, and at end of trial (Expected to reach between 12 - 72 months) ]
  • Number and frequency of Medical Events of Special Interest [ Time Frame: When the first 20 PUPs have reached at least 50 EDs, when the first 40 PUPs have reached 100 EDs, and at end of trial (Expected to reach between 12 - 72 months) ]
  • Number of breakthrough bleeding episodes during prophylaxis (annualised bleeding rate) Haemostatic effect by 4-point haemostatic response scale ("excellent", "good", "moderate" and "poor") [ Time Frame: When the first 20 PUPs have reached at least 50 EDs, when the first 40 PUPs have reached 100 EDs, and at end of trial (Expected to reach between 12 - 72 months) ]
  • Haemostatic effect by 4-point haemostatic response scale ("excellent", "good", "moderate" and "poor") [ Time Frame: When the first 20 PUPs have reached at least 50 EDs, when the first 40 PUPs have reached 100 EDs, and at end of trial (Expected to reach between 12 - 72 months) ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Safety and Efficacy of Nonacog Beta Pegol (N9-GP) in Previously Untreated Patients With Haemophilia B
Official Title  ICMJE An Open-label Single-arm Multicentre Non-controlled Phase 3 a Trial Investigating Safety and Efficacy of Nonacog Beta Pegol (N9-GP) in Prophylaxis and Treatment of Bleeding Episodes in Previously Untreated Patients With Haemophilia B (FIX Activity Below or Equal to 2 Percent)
Brief Summary This trial is conducted globally. The aim of the trial is to investigate the safety and efficacy of nonacog beta pegol (N9-GP) in previously untreated patients with Haemophilia B.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Congenital Bleeding Disorder
  • Haemophilia B
Intervention  ICMJE Drug: nonacog beta pegol
For intravenous (i.v.) injection. A single dose of 40 U/kg, unless the bleeding episode is severe in which case it should be treated with 80 U/kg.
Study Arms  ICMJE Experimental: 50 EDs (exposure days)
Intervention: Drug: nonacog beta pegol
Publications * Chan AK, Alamelu J, Barnes C, Chuansumrit A, Garly ML, Meldgaard RM, Young G. Nonacog beta pegol (N9-GP) in hemophilia B: First report on safety and efficacy in previously untreated and minimally treated patients. Res Pract Thromb Haemost. 2020 Jul 29;4(7):1101-1113. doi: 10.1002/rth2.12412. eCollection 2020 Oct.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Active, not recruiting
Estimated Enrollment  ICMJE
 (submitted: November 15, 2019)
60
Original Estimated Enrollment  ICMJE
 (submitted: May 16, 2014)
50
Estimated Study Completion Date  ICMJE October 30, 2022
Estimated Primary Completion Date October 30, 2022   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Informed consent obtained before any trial-related activities. Trial-related activities are any procedures that are carried out as part of the trial, including activities to determine suitability for the trial
  • Male, age below 6 years at the time of signing informed consent
  • Patients with the diagnosis of haemophilia B (FIX (coagulation factor IX) activity level below or equal to 2%) based on medical records or central laboratory results
  • Previously untreated or exposed to FIX containing products less than or equal to 3 exposure days (5 previous exposures to blood components is acceptable)

Exclusion Criteria:

  • Any history of FIX inhibitors (defined by medical records)
  • Known or suspected hypersensitivity to trial product or related products
  • Previous participation in this trial. Participation is defined as first dose administered of trial product
  • Receipt of any investigational medicinal product within 30 days before screening
  • Congenital or acquired coagulation disorder other than haemophilia B
  • Any chronic disorder or severe disease which, in the opinion of the Investigator, might jeopardise the patient's safety or compliance with the protocol
  • Patient's parent(s)/LAR(s) (legally acceptable representative) mental incapacity, unwillingness to cooperate, or a language barrier precluding adequate understanding and cooperation
Sex/Gender  ICMJE
Sexes Eligible for Study: Male
Ages  ICMJE up to 6 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Algeria,   Argentina,   Australia,   Austria,   Canada,   France,   Germany,   Israel,   Italy,   Japan,   Malaysia,   Netherlands,   Spain,   Taiwan,   Thailand,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02141074
Other Study ID Numbers  ICMJE NN7999-3895
2012-004867-38 ( EudraCT Number )
U1111-1135-9557 ( Other Identifier: WHO )
JapicCTI-142611 ( Registry Identifier: JAPIC )
NL53683.091.15 ( Other Identifier: CCMO )
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Novo Nordisk A/S
Study Sponsor  ICMJE Novo Nordisk A/S
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Global Clinical Registry (GCR, 1452) Novo Nordisk A/S
PRS Account Novo Nordisk A/S
Verification Date November 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP